Programmed cell death protein-1 (PD-1)-targeted immunotherapy is safe and effective in patients with advanced intrahepatic cholangiocarcinoma (iCCA), according to a new retrospective analysis published in Annals of Medicine.

In a cohort of 42 patients with iCCA who received PD-1-targeted immunotherapy, the overall response rate was 23.8%, with 4 and 6 patients achieving complete and partial responses, respectively. The disease control rate was 85.7%, with 26 patients showing stable disease at follow-up radiological evaluation.

Less than half (45.2%) of the patients receiving PD-1-targeted immunotherapy showed radiological disease progression. The median time to tumor progression was 11.6 (95% CI, 7.9-15.3) months.

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Twelve patients died during follow-up. The median overall survival (OS) was 19.3 (95% CI, 14.9-23.7) months, while progression-free survival (PFS) was 11.6 (95% CI, 7.9-15.3) months. Patients with progressive disease had lower median OS (3.1 vs 19.3 months) and PFS (1.9 vs 13.6 months) than patients with nonprogressive disease.

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Moreover, patients treated with PD-1 monotherapy had a significantly shorter median OS than patients receiving combination therapy (11.6  vs 21.3), such as transcatheter arterial chemoembolization and hepatic artery infusion chemotherapy. The median PFS was similar between the 2 groups (10.6 vs 11.5, respectively).

“From the perspective of the trend, the combined treatment has a better efficacy,” the authors said. However, they were not able to further compare combination therapies due to the small sample size.

Half of the patients experienced at least 1 adverse event, with the most common as pain (n=6), anorexia (n=4), hypertension (n=4), pyrexia (n=3), cough (n=3), and hypothyroidism (n=3). Two patients reported grade ≥3 events (pain and hepatitis). In the case of the patient who developed hepatitis, PD-1 immunotherapy was discontinued and corticosteroids were initiated.

The authors also investigated the capacity of the albumin-bilirubin (ALBI) grade to predict PD-1 immunotherapy efficacy in patients with iCCA. Patients with ALBI grade 1 had longer median OS than patients with ALBI grade 2 (19.3 vs 14.7 months). No significant difference was observed for median PFS.


Deng M, Li S, Wang Q, et al. Real-world outcomes of patients with advanced intrahepatic cholangiocarcinoma treated with programmed cell death protein-1-targeted immunotherapy. Ann Med. 2022;54(1):803-811. doi:10.1080/07853890.2022.2048416